UPADACITINIB

The PBAC recommended listing upadacitinib (Rinvoq®) on the PBS for the treatment of complex refractory fistulising Crohn’s disease (FCD). 
The PBAC welcomed the input from health professionals and organisations that supported the listing and acknowledged fistulising disease is a severe and debilitating complication of Crohn’s disease that has a profound impact on patients and there remains a clinical need for new and effective therapies to help patients manage their condition, as many patients do not respond to or lose response to current treatments.
The PBAC considered the clinical evidence presented was uncertain as the evidence base was largely based on small groups of patients with FCD from larger Crohn’s disease trials. However, the PBAC acknowledged the treatments it had previously recommended for FCD were supported by similarly uncertain evidence. On balance, the PBAC was satisfied that upadacitinib was likely to be as effective and safe as the other PBS listed therapies for FCD, including infliximab, adalimumab and ustekinumab. 
The PBAC advised the cost of upadacitinib would be acceptable if the overall cost per patient was not higher than the least costly PBS listed therapy for FCD, and the listing would likely be cost neutral or result in a small cost saving to the PBS if listed on that basis. 
The PBAC advised the restrictions should align with the other PBS listed therapies for FCD including aligning with its recommendation for the broader inflammatory bowel diseases restriction changes made at the same meeting. The PBAC recommended an additional listing for the 30 mg form of upadacitinib for induction therapy in FCD, severe Crohn’s disease and ulcerative colitis for patients with severe renal impairment (as the standard 45 mg initial dose is not appropriate for these patients).